Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of SMA

Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of SMA

Press Release | February 18, 2011

Dr. Charlotte Sumner and colleagues at Johns Hopkins University publish a study in the journal Human Molecular Genetics showing improvement in survival but not motor function in a severe model of Spinal Muscular Atrophy by increasing the levels of the muscle modulator Insulin-like growth factor 1 (IGF-1).